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Phase 2 N=352 Randomized Triple-blind Treatment

A Clinical Study in Participants With Huntington's Disease (HD) to Assess Efficacy and Safety of Three Oral Doses of Laquinimod

Huntington's Disease

Enrolled (actual)
352
Serious AEs
6.0%
Results posted
Jun 2019
Primary outcome: Primary: Change From Baseline in UHDRS-TMS at Week 52 — 1.3; 1.4; 2.0; 11.0 units on a scale — p=0.4853

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Laquinimod (Drug); Placebo (Drug)
Age
Adult · 21+ yrs
Sex
All
Sponsor
Teva Branded Pharmaceutical Products R&D, Inc.
Primary completion
Jun 2018

Outcome Measures

OutcomeResultp-value
PRIMARY
Change From Baseline in UHDRS-TMS at Week 52
1.3; 1.4; 2.0; 11.0 0.4853
SECONDARY
Percent Change From Baseline in Caudate Volume (Brain Atrophy) at Week 52
5.13; 4.03; 3.14; 4.11

Summary

The primary objective of this study is to assess the efficacy of laquinimod as treatment in participants with HD after 52 weeks using the Unified Huntington's Disease Rating Scale Total Motor Score (UHDRS-TMS or TMS).

Eligibility Criteria

Inclusion Criteria

  • Documentation of prior positive genetic testing for HD, or a clinical diagnosis of symptomatic HD.
  • Presence of 36-49 cytosine-adenosine-guanine (CAG) repeats, inclusive, in the huntingtin gene based on centralized CAG testing during screening.
  • Male or female between 21-55 years of age, inclusive, with an onset of HD at or after 18 years of age.
  • Women of child-bearing potential (women who are not post menopausal or who have undergone surgical sterilization) must practice an acceptable method of birth control for 30 days before taking the study treatment, and 2 acceptable methods of birth control during all study duration and until 30 days after the last dose of treatment was administered.
  • A sum of greater than (>) 5 points on the UHDRS-TMS at the screening visit.
  • Able and willing to provide written informed consent prior to any study related procedure being performed at the screening visit. Participants with a legal guardian should be consented according to local requirements.
  • Willing to provide a blood sample for genomic CAG analysis at the screening visit.
  • Willing and able to take oral medication and able to comply with the study specific procedures.
  • Ambulatory, being able to travel to the study center, and judged by the investigator as likely to be able to continue to travel for the duration of the study.
  • Availability and willingness of a caregiver, informant, or family member to provide input at study visits assessing Clinician's Interview-Based Impression of Change (CIBIC)-Plus, Clinical Dementia Rating - Sum of Boxes (CDR-SB), Problem Behaviors Assessment-Short form (PBA-s) and Huntington's Disease Quality of Life (HD-QoL). A caregiver is recommended to be someone who attends to the participant at least 2 to 3 times per week for at least 3 hours per occasion, and the suitability of the caregiver should be judged by the investigator.
  • For participants taking allowed antidepressant medication, the dosing of medication must have been kept constant for at least 30 days before baseline and must be kept constant during the study.
  • Additional criteria may apply, please contact the investigator for more information.

Exclusion Criteria

  • Use of immunosuppressive agents, or cytotoxic agents, including cyclophosphamide and azatioprine within 12 months prior to screening.
  • Previous use of laquinimod.
  • Use of moderate/strong inhibitors of cytochrome P450 (CYP)3A4 within 2 weeks prior to randomization.
  • Use of inducers of CYP3A4 within 2 weeks prior to randomization.
  • Pregnant or breastfeeding.
  • Participants with a clinically significant or unstable medical or surgical condition that may put the participant at risk when participating in the study or may influence the results of the study or affect the participant's ability to take part in the study, as determined by medical history, physical examinations, electrocardiogram (ECG), or laboratory tests. Such conditions may include:
  • A major cardiovascular event (for example; myocardial infarction, acute coronary syndrome, de-compensated congestive heart failure, pulmonary embolism, coronary revascularization) that occurred prior to randomization.
  • Any acute pulmonary disorder.
  • A central nervous system (CNS) disorder other than HD that may jeopardize the participant's participation in the study, including such disorders that are demonstrated on the baseline MRI (based on local read).
  • A gastrointestinal disorder that may affect the absorption of study medication.
  • Acute or chronic renal disease including acute kidney injury (AKI).
  • Any form of acute or chronic liver disease.
  • Known human immunodeficiency virus (HIV) positive status. Participants will undergo an HIV test at screening per local requirements, if applicable.
  • Any malignancies, excluding basal cell carcinoma, in the 5 years prior to randomization.
  • Any clinically significant, abnormal, screening laboratory result which in the opinion of the investigator, affects the
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02215616). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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